US-based Lumena Pharmaceuticals has announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued positive opinions for four orphan drug designations for the company’s lead drug candidate, LUM001.
The COMP has granted first positive opinion for LUM001 for primary sclerosing cholangitis and three positive opinions for three other rare cholestatic liver diseases that include Alagille syndrome, progressive familial intrahepatic cholestasis, and primary biliary cirrhosis.
These four positive opinions follow the orphan drug designation issued by the US Food and Drug Administration (FDA) Office of Orphan Product Development in September for the same indications.
Cholestasis is an impairment of bile formation and bile ?ow in the liver that may lead to progressive liver damage causing liver failure.
LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter that is responsible for recycling bile acids back to the liver.
It is a once-daily oral drug developed to reduce elevated serum bile-acid levels, preventing liver damage and alleviating severe itching.
The drug has been studied in more than 1,400 patients in 12 different clinical studies and the studies have demonstrated that it can reduce serum bile acid levels.
According to the company, the most common side effect with LUM001 is gastrointestinal disturbance.
LUM001 is currently being evaluated by Lumena Pharmaceuticals in a Phase II study in children with Alagille syndrome, and in a Phase II study in adults with progressive familial intrahepatic cholestasis.
The company plans to begin a Phase II study for LUM001 in children with progressive familial intrahepatic cholestasis, and a Phase II study in adults with primary sclerosing cholangitis later in 2013.
Lumena Pharmaceuticals president and CEO Mike Grey said: "We will continue to work diligently to develop LUM001 for the patients in serious need of safe, non-invasive therapeutic options to prevent progressive liver damage and the accompanying devastating symptoms."
The positive opinion of the COMP for LUM001 has been forwarded to the EU commission for final approval.